Briefs · Frontier Science
Two Rare-Disease Approvals Landed on the Same Friday
Two rare-disease approvals landed on the same Friday. The quieter one may matter more to the children it reaches.
The FDA approved an expansion of Joenja (leniolisib) on 11 September, extending it to children aged 4 to 11 with activated PI3Kδ syndrome (APDS) who weigh at least 27 kg — the first FDA-approved treatment for that age group. The oral PI3Kδ inhibitor was previously approved only for patients 12 and older, back in March 2023.
The new 40 mg and 50 mg twice-daily doses are expected to reach patients in October. A separate application, filed in July, seeks lower doses for children weighing 13 to 27 kg — the next rung down the age ladder.
Conclusion: Joenja is not a new drug, but rather a widening door — and for children with a rare immune disorder, the widening is the news.
Read the original at Pharming Group
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